Novartis’ Gene Therapy Shows Promise in Treating SMA

Novartis ( (NVS) ) has provided an update.

Novartis announced the success of its Phase III STEER study for investigational intrathecal onasemnogene abeparvovec (OAV101 IT) in treating spinal muscular atrophy (SMA) Type 2 in children and young adults. The study demonstrated an increase in motor function, showcasing the therapy’s efficacy and safety compared to a sham control. Novartis plans to seek regulatory approval in 2025, which could enhance treatment options for SMA patients and strengthen the company’s position in gene therapy innovation.

More about Novartis

Novartis AG is a global healthcare company based in Basel, Switzerland, specializing in innovative medicines, eye care products, and generic pharmaceuticals. The company focuses on the development of treatments for various diseases, leveraging its expertise in biology and technology to address unmet medical needs.

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